Meet Damali and Amiel Reid, two siblings who are living with sickle cell anemia, and learn about their struggles with the disease and their hopes for a cure.

Sickle cell anemia is a genetic blood disorder affecting an estimated 70,000 to 100,000 people in the United States. In the disorder, hemoglobin – an important protein that is present in red blood cells and carries oxygen throughout the body – is abnormal, causing the red blood cells to bend into a sickle-like shape. These oddly-shaped cells clump together, getting stuck in blood vessels, resulting in severe pain, infections, damage to organs, and a shortened life expectancy.

At Children’s, our Stem Cell Transplantation Program works in close collaboration with the Dana Farber Cancer Institute to perform hematopoietic stem cell transplants to treat sickle cell anemia and other blood disorders. The Stem Cell Transplantation Program was established in the 1970s, and it is one of the largest and most active pediatric transplantation programs in the United States.

While transplants like these are proven, life-saving options for many patients, they are not a universal solution, or an infallible one. Compatible donors cannot always be found, and even when a matching donor is found, there is a risk of the patient’s body rejecting the transplant. George Q. Daley, Director of the Stem Cell Transplantation Program, sees these problems in the clinic; his research laboratory and others at Children’s are working on ways to overcome them.

Dr. Daley’s research includes using stem cell treatments to correct hemoglobin disorders like sickle cell anemia in mouse models.

This research gives patients like Damali and Amiel hope for the future. Your gift can help their hope and our research move closer to a cure.

Spotlight

Blood diseases: The First Frontier

Hematologist George Q. Daley, MD, PhD, Director of Stem Cell Transplantation
Program, has seen children with blood diseases die, often because they aren’t candidates for bone marrow transplants, currently the best tool for treating many of these diseases. Daley hopes to employ pluripotent stem cells to create safer, genetically matched bone-marrow transplants for patients. Read more.

Stem cell therapy for blood diseases

In this video clip, Leonard Zon, MD, discusses why stem cell therapy for blood diseases will likely make it to the bedside first. He then goes on to discuss an exciting new treatment—now in clinical trial—that promises to make this therapy more effective and efficient.